CDNF Phase 1 Trial - PubMed
Huttunen et al./PubMed/2023
Why It Matters
This caught my attention because it's testing a fundamentally different approach to Parkinson's — not managing symptoms, but potentially protecting the dopamine neurons that die in the disease. The preclinical data looked promising, but this is first-in-human testing focused purely on safety. No efficacy data yet, and the delivery method (brain surgery plus implanted catheters) is invasive. Worth watching, but years away from knowing if it actually helps patients.
Key Findings
- 17 Parkinson's patients received CDNF delivered directly into the putamen (deep brain structure) through surgically implanted catheters over 6 months
- Treatment was well-tolerated with no CDNF-related serious adverse events, though surgical complications (infections, hardware issues) occurred as expected with brain surgery
- This was a phase 1 safety trial — no efficacy endpoints were measured, so we don't know yet if CDNF actually improves motor function or slows disease progression in humans
- CDNF works differently than conventional growth factors — it stays inside cells and helps with protein folding and ER stress, which may protect dopamine neurons
- The delivery method is a significant barrier: requires neurosurgery, implanted hardware, and repeated infusions — far from a practical treatment even if it works
Read the Paper↗PMID: 37212361